Mechanism underlying so-called idiopathic autism inception unveiled
Investigators at the Institute for Research in Biomedicine (IRB Barcelona) have unraveled how and why the absence of a neuronal microexon in cytoplasmic polyadenylation element-binding 4 (CPEB4) gives rise to autism. In 2018, investigators from IRB, co-led by Raúl Méndez, identified the overt correlation between defects in CPEB4 and the onset of autism. However, the previous work did not provide the molecular mechanism explaining the correlation.

Mechanism underlying so-called idiopathic autism inception unveiled
Investigators at the Institute for Research in Biomedicine (IRB Barcelona) have unraveled how and why the absence of a neuronal microexon in cytoplasmic polyadenylation element-binding 4 (CPEB4) gives rise to autism. In 2018, investigators from IRB, co-led by Raúl Méndez, identified the overt correlation between defects in CPEB4 and the onset of autism. However, the previous work did not provide the molecular mechanism explaining the correlation.
UB-VV111 shows favorable safety, low risk of off-target transduction
To address the existing challenges with CAR T-cell therapy, scientists at Umoja Biopharma Inc. developed the Vivovec platform, which is an off-the-shelf surface-engineered lentiviral vector (LVV) drug product designed to generate CAR T cells in vivo without lymphodepletion by selectively binding, activating and transducing T cells.
Peptidream announces new peptide radiopharmaceutical development candidate targeting CLDN12.8
Peptidream Inc. has announced a new peptide radiopharmaceutical development candidate, PD-29875, a novel first-in-class highly selective macrocyclic peptide-radioisotope conjugate against Claudin 18.2 (CLDN18.2).
OPN-6602 exhibits potency against multiple myeloma in vitro and in vivo
Opna Bio AG has presented promising data regarding their EP300/CBP inhibitor OPN-6602 for the treatment of multiple myeloma (MM). The inhibition of EP300/CBP causes cell cycle arrest and apoptosis in MM cells due to suppression of interferon regulatory factor 4 (IRF4) and MYC repression.
Other news to note for Dec. 13, 2024
Additional early-stage research and drug discovery news in brief.
SK Bioscience’s mRNA Japanese encephalitis vaccine receives clinical trial clearance in Australia
SK Bioscience has received approval from the Human Research Ethics Committee in Australia for a phase I/II trial of its mRNA-based Japanese encephalitis vaccine candidate, GBP-560.
Elevation Oncology nominates HER3 ADC as development candidate
Elevation Oncology Inc. has nominated EO-1022 as its HER3 antibody-drug conjugate (ADC) development candidate for the treatment of solid tumors that express HER3.
Baylor College of Medicine patents new BTK, IKZF1 and IKZF3 degradation inducers
Baylor College of Medicine has disclosed proteolysis targeting chimeras (PROTACs) comprising cereblon (CRBN) ligands coupled to Bruton tyrosine kinase (BTK)-, DNA-binding protein Ikaros (IKZF1)- and Zinc finger protein Aiolos (IKZF3)-targeting moieties through a linker.
Adimune completes preclinical efficacy and safety studies of immune modulation therapy ADI-100
Adimune Inc., a subsidiary of Aditxt Inc., has successfully completed preclinical studies for its antigen-specific gene therapy, ADI-100.

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Conferences

CAP-002 exerts robust and sustained phenotypic correction in genetic epilepsy
Neurology/psychiatric
Epileptic encephalopathy due to mutations in the STXBP1 gene, also known as genetic epilepsy, is a rare disease characterized by intellectual disability, speech and motor impairment and behavioral issues, among others, that affects 1 in every 30,000 newborns and which has no approved therapies to...
PW-507 preferentially reduces binge-like eating in rats
Neurology/psychiatric
Researchers from State University of New Jersey (Rutgers) presented preclinical data for PW-507, a sigma-1 receptor antagonist being evaluated for the treatment of binge eating disorder.
ATL-201, a di-siRNA, reduces seizures in KCNT1-driven epilepsy
Neurology/psychiatric
Pathogenic variants in the KCNT1 gene, which encodes potassium channel subfamily T member 1, cause a severe childhood developmental epileptic encephalopathy.
AU8-18, a new potent oral CDK2 inhibitor with efficacy in ovarian cancer model
New compound
Gynecological cancers with amplifications in the CCNE1 gene, which encodes cyclin E1, usually exhibit resistance to standard therapies. Since cyclin-dependent kinase 2 (CDK2) is the primary partner of cyclin E, CDK2 inhibitors represent a potentially effective treatment strategy for these...

Cancer

Gram-negative bacteria platform offers multiple immune receptor agonists for tumor immunotherapy
Immuno-oncology
Previous research suggested that gram-negative bacteria may hold potential for cancer immunotherapy because they contain agonists for multiple immune receptors.

Infection

INO-4500 provides long-lasting protection against Lassa fever
Vaccine
Researchers from Inovio Pharmaceuticals Inc. described the protective effect exerted by INO-4500, a DNA vaccine produced using the Syncon technology, against lethal Lassa virus in nonhuman primates.

Financing at Orthogon Therapeutics supports development of oral therapy for BK virus
Financings
Orthogon Therapeutics LLC has closed an oversubscribed financing round, with funding to be used to accelerate the development of its first-in-class treatments for managing BK virus reactivation in transplant patients.
miR-1-3p levels predict case severity in hospitalized COVID-19 patients
Biomarkers
Increasing evidence shows that circulating microRNAs (miRNAs) regulate biological processes involved in COVID-19 complications.

Neurology/psychiatric

Preclinical efficacy of M1/M4 receptor agonist in schizophrenia, cognitive impairment models
Conferences
Researchers from Maplight Therapeutics Inc. presented preclinical data for the investigational muscarinic M1/M4 receptor agonist ML-007, being evaluated for the treatment of neuropsychiatric disorders, such as schizophrenia and Alzheimer’s disease (AD). The activity of ML-007 was compared to that...
Engineered T cells can serve as organ-specific delivery service
Science
Researchers from the University of California San Francisco (UCSF) have successfully replicated the design of regulatory T cells, achieving local targeted immune suppression and protection from CAR T-cell cytotoxicity. Many of the treatments used so far in the context of inflammatory and autoimmune...
Dewpoint and Mitsubishi ink $480M ALS research collaboration
Deals and M&A
Dewpoint Therapeutics Inc. and Mitsubishi Tanabe Pharma Corp. have entered a research collaboration worth up to $480 million to advance Dewpoint’s novel TDP-43 small-molecule condensate modulator for amyotrophic lateral sclerosis. Under terms of the deal, Boston-based Dewpoint will receive an...
Equity investment in Eikonizo supports HDAC6 inhibitor work
Cardiovascular
Eikonizo Therapeutics Inc. has secured an equity investment from Novo Nordisk A/S.
BMB-101 5-HT2C biased agonist overcomes tolerance in chronic epilepsy treatment
Conferences
Researchers from Bright Minds Biosciences Inc. presented data on the mechanism of action of BMB-101, a novel scaffold 5-HT2C Gq-protein biased designed to treat drug-resistant neurological disorders.

Endocrine/metabolic

Bio-path to develop BP-1001-A to treat obesity in type 2 diabetes patients
Bio-path Holdings Inc. has outlined its plans to develop BP-1001-A, a drug product modification of prexigebersen.

Biomarkers

Biomarker could aid in psychosis diagnosis
Science
Researchers at the University of Rochester have described a neuroimaging-based biomarker that could identify individuals with early psychosis, and improved their identification when it was added to a standard neurocognitive diagnostic test. In a group of roughly 160 participants in the Human...

Loss-of-function variants linked to neurodevelopmental disorder with hypercholanemia
Genetic/congenital
WD repeat domain 83 opposite strand (WDR83OS) encodes the 106-aa (amino acid) protein Asterix, which is a binding partner for CCDC47. More specifically, Asterix heterodimerizes with CCDC47 to form the protein associated with ER translocon (PAT) complex that specifically chaperones large proteins...
HOXB13 gene involved in metastatic omentum carcinoma, study shows
Conferences
Primary tumors arising in the gastrointestinal system, ovary or pancreas may spread and lead to secondary cancers such as metastatic omentum cancer.

Gastrointestinal

NIDDK grant supports Orphagen’s RARα antagonist for inflammatory bowel disease
Grant
Orphagen Pharmaceuticals Inc. has been granted an award of up to $1.7 million.
Amgen discloses 15-PGDH inhibitors
Patents
Amgen Inc. has reported new 5,6- and 6,6-fused bicyclic alcohols and ethers acting as 15-hydroxyprostaglandin dehydrogenase (15-PGDH) inhibitors. They are reported to be useful for the treatment of gastrointestinal disorders.
SRT-015 prevents acute liver injury in preclinical tests
Conferences
Genfit SA and its collaborators have described the preclinical efficacy of SRT-015, a liver-selective ASK1 inhibitor in models of liver failure.
EA-3571, a dual inhibitor of enteropeptidase and trypsin with efficacy in mouse model of MASH
Conferences
Recently presented preclinical data show that EA Pharma Co. Ltd.'s EA-3571 is a highly potent dual inhibitor of enteropeptidase and trypsin with luminal action, resulting in potential anti-insulin resistance and fat-burning properties.
Orsobio ACMSD inhibitor reverses liver injury in preclinical models
Conferences
The enzyme aminocarboxymuconate semialdehyde decarboxylase (ACMSD) is a regulator of de novo NAD+ synthesis and is reduced in patients with advanced liver disease.
Dual-targeted approach for hepatobiliary diseases presented
Conferences
Rectify Pharmaceuticals Inc. has conducted preclinical testing on RTY-694, an oral dual-targeted positive functional modulator for use in the treatment of hepatobiliary disorders.

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